Background and importance New recombinant long half-life factors VIII (RLHF) have been added to the therapeutic arsenal with the aim of improving the treatment of patients with severe haemophilia A as prophylaxis. After 2 years of treatment, we want to analyse if it has resulted in a real improvement.
Aim and objectives To determine the decrease in the number of infusions, consumption of international units (IU) of factor and how this has influenced spending. We also determined if the change has meant an improvement in adherence to treatment.
Material and methods This was an observational prospective study in a hospital with a reference unit for congenital coagulopathies that included all patients who began treatment with RLHF and had been on treatment for at least 3 months: rurioctocog (Adynovi), lonoctocog (Afstyla) and efmoroctocog (Elocta). Treatment with RLHF was compared with conventional factor VIII (CF) that was administered before the change, during the whole period with RLFH and the whole last period (CF). Adherence, number of infusions, IU consumed and cost/month were compared. Adherence was calculated considering the number of IU dispensed at the pharmacy and the number of IU prescribed. Changes >10% were considered relevant. Adherence values >100% were treated as 100%. Microsoft Office Access and Excel were used for the recording of variables and statistical analysis.
Results Thirty-five patients were included, all men, with a median age of 19 (ICR 12–28) years; all patients had previously received recombinant factor VIII except for two patients who had received plasmatic factor. We found that 31% of patients improved their adherence by more than 10% by switching to RLHF: 14% of patients reduced their adherences by >10% and 55% of patients maintained their adherence. Patients with <90% adherence with the previous treatment was 37% and with RLHF was 22%. Median monthly infusions were 12 and a median of 2 monthly infusions was reduced by switching to RLHF. The median number of IU saved per patient/month was 7000 (ICR (−8000); 1000) IU. This resulted in a median savings per patient/month of 3182 (ICR (−3.654); (−5))€.
Conclusion and relevance RLHF is a discrete advance in haemophilia therapy and it decreased the number of infusions/month with a small improvement in adherence. Less IU was consumed/month, and this was a cost saving.
References and/or acknowledgements No conflict of interest.
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